BPX-601

Bellicum was running phase 1/2 clinical trials of two GoCAR-T candidates. Bellicum Pharmaceuticals’ GoCAR-T cell therapies have skidded out, with the biotech halting trials of the two candidates over dose-limiting toxicities. The evaporation of Bellicum’s pipeline prompted the CAR-T cell therapy specialist to start an evaluation of strategic alternatives. Houston-based Bellicum was running phase 1/2 clinical trials of two CAR-T candidates, namely the PSCA prospect BPX-601 and the HER2 candidate BPX-603. Both assets use the biotech’s GoCAR-T technology. BPX-601 expresses a rimiducid-inducible co-activation switch to enhance T cell potency and persistence. And BPX-603 features both the co-activation switch and a CaspaCIDe safety switch. Bellicum pitched the switches as ways to boost effector cell proliferation, enhance functional persistence  and empower physicians to wipe out the CAR-T cells in the event of a serious side effect such as cytokine release syndrome. Yet, the early data on the candidates have discouraged Bellicum. The most recent metastatic castration-resistant prostate cancer patient to receive BPX-601 experienced serious immune-mediated adverse events including grade 4 cytokine release syndrome. The adverse events marked the second time Bellicum has seen a dose-limiting toxicity in that dose escalation cohort. In response, the biotech paused enrollment and reviewed the data. The review showed four of the nine prostate cancer patients experienced a 90% or greater reduction in the PSA biomarker. But also revealed the need to optimize either the clinical dose and schedule of BPX-601 and the activating agent rimiducid, or the design of the BPX-601 cell construct to make the benefits outweigh the risks.  Bellicum, which ended September with $28.8 million to its name, lacks the cash to pull off the BPX-601 rescue mission, leading it to stop clinical trials of the candidate and its other pipeline prospect BPX-603. With all clinical development winding down, the biotech is now evaluating strategic alternatives.   The initiation of the evaluation suggests Bellicum’s drawn-out demise is now nearing the end. Back in 2014, the biotech was one of three CAR-T cell therapy developers to arrive on Nasdaq. The other two cell therapy developers—Kite Pharma and Juno Therapeutics—went on to land multi-billion dollar buyouts and deliver commercial products.  Bellicum has looked unlikely to follow in the footsteps of the other members of the CAR-T class of 2014 for several years. In 2020, the company laid off 79% of its employees after BPX-601 delivered lackluster safety and efficacy data.  

HOUSTON, TX, USA I March 14, 2023 I Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), today announced its decision to discontinue its ongoing Phase 1/2 clinical trials evaluating the safety and preliminary efficacy of its GoCAR-T cell product candidates in combination with rimiducid in heavily pre-treated cancer patients. The trials for BPX-601 and BPX-603 are being discontinued following the Company’s assessment of the risk/benefit profile of BPX-601 in combination with rimiducid. The most recent patient treated in the Phase 1/2 trial of BPX-601 in metastatic castration-resistant prostate cancer (mCRPC) experienced serious immune-mediated adverse events including Grade 4 cytokine release syndrome (CRS), the second dose-limiting toxicity observed in this cohort of dose escalation. The company paused enrollment in its clinical studies and conducted a thorough review of the risk/benefit observed to date. While clinically meaningful efficacy has been observed—including 5 of 9 mCRPC patients treated achieving PSA50 response, 4 of whom achieved PSA90 response—the Company believes it does not have the necessary resources to optimize either the clinical dose and schedule of BPX-601 cells and the activating agent rimiducid, or the design of the BPX-601 cell construct to achieve a favorable risk/benefit profile. The Company is communicating with clinical trial sites and regulatory agencies regarding its decision to discontinue its trials, and an evaluation of the Company’s strategic alternatives is underway. More information about Bellicum can be found at www.bellicum.com . SOURCE: Bellicum Pharmaceuticals